BR112021018776A2 - Recombinant adeno-associated virus vectors - Google Patents

Recombinant adeno-associated virus vectors

Info

Publication number
BR112021018776A2
BR112021018776A2 BR112021018776A BR112021018776A BR112021018776A2 BR 112021018776 A2 BR112021018776 A2 BR 112021018776A2 BR 112021018776 A BR112021018776 A BR 112021018776A BR 112021018776 A BR112021018776 A BR 112021018776A BR 112021018776 A2 BR112021018776 A2 BR 112021018776A2
Authority
BR
Brazil
Prior art keywords
virus vectors
recombinant adeno
associated virus
aav capsid
virus
Prior art date
Application number
BR112021018776A
Other languages
Portuguese (pt)
Inventor
Daniel Mccoy
E Berry Garrett
Original Assignee
Stridebio Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Stridebio Inc filed Critical Stridebio Inc
Publication of BR112021018776A2 publication Critical patent/BR112021018776A2/en

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14145—Special targeting system for viral vectors

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Virology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biophysics (AREA)
  • Medicinal Chemistry (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Plant Pathology (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Epidemiology (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Peptides Or Proteins (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

vetores de vírus adenoassociado recombinante. as proteínas de capsídeo de aav compreendendo uma modificação na sequência de aminoácidos e vetores de vírus compreendendo a proteína de capsídeo de aav modificada são descritas. também são descritos métodos de administração de vetores de vírus e capsídeos de vírus a uma célula ou a um sujeito in vivo.recombinant adeno-associated virus vectors. aav capsid proteins comprising a modification in the amino acid sequence and virus vectors comprising the modified aav capsid protein are described. Methods of administering virus vectors and virus capsids to a cell or subject in vivo are also described.

BR112021018776A 2019-03-21 2020-03-20 Recombinant adeno-associated virus vectors BR112021018776A2 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201962821710P 2019-03-21 2019-03-21
PCT/US2020/023877 WO2020191300A1 (en) 2019-03-21 2020-03-20 Recombinant adeno-associated virus vectors

Publications (1)

Publication Number Publication Date
BR112021018776A2 true BR112021018776A2 (en) 2021-11-30

Family

ID=70289855

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112021018776A BR112021018776A2 (en) 2019-03-21 2020-03-20 Recombinant adeno-associated virus vectors

Country Status (16)

Country Link
US (2) US20220064675A1 (en)
EP (1) EP3941929A1 (en)
JP (1) JP2022525955A (en)
KR (1) KR20220011616A (en)
CN (1) CN113727992A (en)
AR (1) AR118465A1 (en)
AU (1) AU2020241888A1 (en)
BR (1) BR112021018776A2 (en)
CA (1) CA3133453A1 (en)
CO (1) CO2021013878A2 (en)
EA (1) EA202192501A1 (en)
IL (1) IL286464A (en)
MX (1) MX2021011468A (en)
SG (1) SG11202110146VA (en)
TW (1) TW202102525A (en)
WO (1) WO2020191300A1 (en)

Families Citing this family (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3774854A1 (en) 2018-04-03 2021-02-17 Stridebio, Inc. Antibody-evading virus vectors
EA202192501A1 (en) 2019-03-21 2021-12-16 Страйдбайо, Инк. RECOMBINANT ADENO-ASSOCIATED VIRAL VECTORS
WO2021076925A1 (en) 2019-10-17 2021-04-22 Stridebio, Inc. Adeno-associated viral vectors for treatment of niemann-pick disease type c
KR20220139956A (en) 2020-02-13 2022-10-17 테나야 테라퓨틱스, 인코포레이티드 Gene therapy vectors to treat heart disease
WO2021163357A2 (en) 2020-02-13 2021-08-19 Tenaya Therapeutics, Inc. Gene therapy vectors for treating heart disease
TW202208397A (en) 2020-05-13 2022-03-01 美商航海家醫療公司 Redirection of tropism of aav capsids
CN113025658B (en) * 2021-03-10 2022-04-22 华侨大学 Delivery vector of neural stem cell specific gene and application thereof
CN112961220B (en) * 2021-04-19 2023-11-17 信念医药科技(上海)有限公司 Adeno-associated virus capsid protein and adeno-associated virus vector comprising same
US20230133924A1 (en) 2021-07-08 2023-05-04 Tenaya Therapeutics, Inc. Optimized expression cassettes for gene therapy
WO2023150687A1 (en) * 2022-02-04 2023-08-10 Ginkgo Bioworks, Inc. Recombinant adeno-associated virus vectors, and methods of use thereof
US20230357434A1 (en) * 2022-03-03 2023-11-09 California Institute Of Technology Compositions and methods for crossing blood brain barrier
KR102578015B1 (en) 2022-07-18 2023-09-18 주식회사 마크헬츠 An AAV vector containing a promoter specifically expressed in cerebrovascular endothelial cells and coated with a nano-peptide for targeting cerebrovascular endothelial cells, and its use for cerebrovascular endothelial cell-specific gene delivery
WO2024054864A1 (en) 2022-09-06 2024-03-14 Tenaya Therapeutics, Inc. Cardioprotective heart disease therapies

Family Cites Families (254)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4039388A (en) 1976-06-04 1977-08-02 The United States Of America As Represented By The Government Diagnostic test for Niemann-Pick disease
US4501729A (en) 1982-12-13 1985-02-26 Research Corporation Aerosolized amiloride treatment of retained pulmonary secretions
US5139941A (en) 1985-10-31 1992-08-18 University Of Florida Research Foundation, Inc. AAV transduction vectors
EP0322417A1 (en) 1986-09-08 1989-07-05 Applied Biotechnology, Inc. Empty viral capsid vaccines
US4968603A (en) 1986-12-31 1990-11-06 The Regents Of The University Of California Determination of status in neoplastic disease
JPH04501719A (en) 1988-11-10 1992-03-26 インペリアル・キヤンサー・リサーチ・テクノロジー・リミテツド polypeptide
US5916563A (en) 1988-11-14 1999-06-29 United States Of America Parvovirus protein presenting capsids
US5328470A (en) 1989-03-31 1994-07-12 The Regents Of The University Of Michigan Treatment of diseases by site-specific instillation of cells or site-specific transformation of cells and kits therefor
US5399346A (en) 1989-06-14 1995-03-21 The United States Of America As Represented By The Department Of Health And Human Services Gene therapy
ES2026826A6 (en) 1991-03-26 1992-05-01 Ercros Sa Method for producing a subunit vaccine against the canine parvovirus and other related viruses.
US5773278A (en) 1991-05-03 1998-06-30 Mount Sinai Medical Center Acid sphingomyelinase gene
US5858784A (en) 1991-12-17 1999-01-12 The Regents Of The University Of California Expression of cloned genes in the lung by aerosol- and liposome-based delivery
US5478745A (en) 1992-12-04 1995-12-26 University Of Pittsburgh Recombinant viral vector system
DK0733103T3 (en) 1993-11-09 2004-07-12 Univ Johns Hopkins Preparation of High Titers of Recombinant AAV Vectors
US5869248A (en) 1994-03-07 1999-02-09 Yale University Targeted cleavage of RNA using ribonuclease P targeting and cleavage sequences
EP0755454B1 (en) 1994-04-13 2008-02-13 The Rockefeller University Aav-mediated delivery of dna to cells of the nervous system
US6204059B1 (en) 1994-06-30 2001-03-20 University Of Pittsburgh AAV capsid vehicles for molecular transfer
US5599706A (en) 1994-09-23 1997-02-04 Stinchcomb; Dan T. Ribozymes targeted to apo(a) mRNA
US6093570A (en) 1995-06-07 2000-07-25 The University Of North Carolina At Chapel Hill Helper virus-free AAV production
ATE452981T1 (en) 1995-06-07 2010-01-15 Univ North Carolina TRANSDUCTION OF MYOBLASTS USING VECTORS FROM ADENO-ASSOCIATED VIRUSES
US6040183A (en) 1995-06-07 2000-03-21 University Of North Carloina At Chapel Hill Helper virus-free AAV production
US6083702A (en) 1995-12-15 2000-07-04 Intronn Holdings Llc Methods and compositions for use in spliceosome mediated RNA trans-splicing
US6013487A (en) 1995-12-15 2000-01-11 Mitchell; Lloyd G. Chimeric RNA molecules generated by trans-splicing
US5962313A (en) 1996-01-18 1999-10-05 Avigen, Inc. Adeno-associated virus vectors comprising a gene encoding a lyosomal enzyme
CA2265460A1 (en) 1996-09-11 1998-03-19 The Government Of The United States Of America, Represented By The Secre Tary, Department Of Health And Human Services Aav4 vector and uses thereof
US6156303A (en) 1997-06-11 2000-12-05 University Of Washington Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom
US6426198B1 (en) 1997-07-03 2002-07-30 The United States Of America As Represented By The Secretary Of The Department Of Health And Human Services Genes for Niemann-Pick type C disease
US6984517B1 (en) 1998-05-28 2006-01-10 The United States Of America As Represented By The Department Of Health And Human Services AAV5 vector and uses thereof
CA2329060C (en) 1998-05-28 2011-09-13 John A. Chiorini Aav5 vector and uses thereof
US6562958B1 (en) 1998-06-09 2003-05-13 Genome Therapeutics Corporation Nucleic acid and amino acid sequences relating to Acinetobacter baumannii for diagnostics and therapeutics
US8299321B2 (en) 1998-06-16 2012-10-30 Monsanto Technology Llc Nucleic acid molecules and other molecules associated with plants and uses thereof for plant improvement
US20100293663A2 (en) 1998-06-16 2010-11-18 Thomas La Rosa Nucleic Acid Molecules and Other Molecules Associated with Plants and Uses Thereof for Plant Improvement
US6544785B1 (en) 1998-09-14 2003-04-08 Mount Sinai School Of Medicine Of New York University Helper-free rescue of recombinant negative strand RNA viruses
EP1115880A2 (en) 1998-09-22 2001-07-18 University of Florida Methods for large-scale production of recombinant aav vectors
GB9822763D0 (en) 1998-10-20 1998-12-16 Univ Sheffield Immunoglobin variant
EP1127150B1 (en) 1998-11-05 2007-05-16 The Trustees Of The University Of Pennsylvania Adeno-associated virus serotype 1 nucleic acid sequences, vectors and host cells containing same
US6759237B1 (en) 1998-11-05 2004-07-06 The Trustees Of The University Of Pennsylvania Adeno-associated virus serotype 1 nucleic acid sequences, vectors and host cells containing same
US6491907B1 (en) 1998-11-10 2002-12-10 The University Of North Carolina At Chapel Hill Recombinant parvovirus vectors and method of making
US6822071B1 (en) 1998-11-12 2004-11-23 The Regents Of The University Of California Polypeptides from Chlamydia pneumoniae and their use in the diagnosis, prevention and treatment of disease
EP1887081A2 (en) 1999-02-25 2008-02-13 Ceres Incorporated DNA Sequences
EP1033405A3 (en) 1999-02-25 2001-08-01 Ceres Incorporated Sequence-determined DNA fragments and corresponding polypeptides encoded thereby
US20110093981A9 (en) 1999-05-06 2011-04-21 La Rosa Thomas J Nucleic acid molecules and other molecules associated with transcription in plants and uses thereof for plant improvement
US20080229439A1 (en) 1999-05-06 2008-09-18 La Rosa Thomas J Nucleic acid molecules and other molecules associated with transcription in plants and uses thereof for plant improvement
US20040031072A1 (en) 1999-05-06 2004-02-12 La Rosa Thomas J. Soy nucleic acid molecules and other molecules associated with transcription plants and uses thereof for plant improvement
US20130326723A1 (en) 1999-05-06 2013-12-05 Thomas J. La Rosa Soy nucleic acid molecules and other molecules associated with plants and uses thereof for plant improvement
US20090087878A9 (en) 1999-05-06 2009-04-02 La Rosa Thomas J Nucleic acid molecules associated with plants
US20110214206A1 (en) 1999-05-06 2011-09-01 La Rosa Thomas J Nucleic acid molecules and other molecules associated with plants
US6498244B1 (en) 1999-05-28 2002-12-24 Cell Genesys, Inc. Adeno-associated virus capsid immunologic determinants
US7314912B1 (en) 1999-06-21 2008-01-01 Medigene Aktiengesellschaft AAv scleroprotein, production and use thereof
DE19933288A1 (en) 1999-07-15 2001-01-18 Medigene Ag Structural protein of adeno-associated virus with altered antigenicity, its production and use
DK1916258T3 (en) 1999-08-09 2014-07-28 Genzyme Corp Increasing expression of a single-stranded heterologous nucleotide sequence from recombinant viral vectors in such a design of the sequence to form intra-strand base pairs
AU2138601A (en) 1999-12-22 2001-07-03 Aventis Pasteur Limited Chlamydia antigens and corresponding DNA fragments and uses thereof
US6468524B1 (en) 2000-03-22 2002-10-22 The United States Of America, As Represented By The Secretary Of The Department Of Health And Human Services AAV4 vector and uses thereof
US20110131679A2 (en) 2000-04-19 2011-06-02 Thomas La Rosa Rice Nucleic Acid Molecules and Other Molecules Associated with Plants and Uses Thereof for Plant Improvement
US20140130203A1 (en) 2000-04-19 2014-05-08 Thomas J. La Rosa Rice nucleic acid molecules and other molecules associated with plants and uses thereof for plant improvement
WO2001081581A2 (en) 2000-04-21 2001-11-01 Corixa Corporation Compositions and methods for the therapy and diagnosis of acne vulgaris
US7465583B2 (en) 2000-06-01 2008-12-16 The University Of North Carolina At Chapel Hill Duplexed parvovirus vectors
US7201898B2 (en) 2000-06-01 2007-04-10 The University Of North Carolina At Chapel Hill Methods and compounds for controlled release of recombinant parvovirus vectors
US7214786B2 (en) 2000-12-14 2007-05-08 Kovalic David K Nucleic acid molecules and other molecules associated with plants and uses thereof for plant improvement
US7749492B2 (en) 2001-01-05 2010-07-06 Nationwide Children's Hospital, Inc. AAV vectors and methods
WO2002053703A2 (en) 2001-01-05 2002-07-11 Children's Hospital, Inc. Aav2 vectors and methods
CA2445969A1 (en) 2001-04-30 2002-11-07 Cell Genesys, Inc. Viral-mediated delivery and in vivo expression of polynucleotides encoding anti-angiogenic proteins
AU2002341541A1 (en) 2001-06-22 2003-03-03 Syngenta Participations Ag Abiotic stress responsive polynucleotides and polypeptides
US7777097B2 (en) 2001-06-22 2010-08-17 Syngenta Participations Ag Plant disease resistance genes
US6623729B2 (en) 2001-07-09 2003-09-23 Korea Advanced Institute Of Science And Technology Process for preparing sustained release micelle employing conjugate of anticancer drug and biodegradable polymer
AU2001289843A1 (en) 2001-08-28 2002-02-13 Bayer Cropscience Ag Polypeptides for identifying herbicidally active compounds
WO2003033515A1 (en) 2001-10-15 2003-04-24 Corixa Corporation Compositions and methods for the therapy and diagnosis of acne vulgaris
WO2003042361A2 (en) 2001-11-09 2003-05-22 Government Of The United States Of America, Department Of Health And Human Services Production of adeno-associated virus in insect cells
CA2406745C (en) 2001-11-13 2006-01-10 The Trustees Of The University Of Pennsylvania A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby
FI3517134T3 (en) 2001-12-17 2024-04-03 Univ Pennsylvania Adeno-associated virus (aav) serotype 8 sequences, vectors containing same and uses therefor
EP3339430A1 (en) 2001-12-17 2018-06-27 The Trustees of The University of Pennsylvania Adeno-associated virus (aav) serotype 9 sequences, vectors containing same, and uses thereof
WO2003093295A2 (en) 2002-04-30 2003-11-13 University Of North Carolina At Chapel Hill Secretion signal vectors
NZ561656A (en) 2002-05-01 2009-03-31 Univ Florida Improved rAAV expression systems for genetic modification of specific capsid proteins
ITRM20020253A1 (en) 2002-05-08 2003-11-10 Univ Roma SNRNA CHEMICAL MOLECULES WITH ANTISENSE SEQUENCES FOR SPLICING JUNCTIONS OF THE DYSTROPHINE GENE AND THERAPEUTIC APPLICATIONS.
JP2005185101A (en) 2002-05-30 2005-07-14 National Institute Of Agrobiological Sciences VEGETABLE FULL-LENGTH cDNA AND UTILIZATION THEREOF
US20070015238A1 (en) 2002-06-05 2007-01-18 Snyder Richard O Production of pseudotyped recombinant AAV virions
WO2004058990A2 (en) 2002-12-20 2004-07-15 Applera Corporation Genetic polymorphisms associated with stenosis, methods of detection and uses thereof
PL215171B1 (en) 2002-12-24 2013-10-31 Rinat Neuroscience Corp Anti-ngf antibodies and methods using same
AU2004248136B2 (en) 2003-06-02 2011-09-15 University Of Massachusetts Methods and compositions for controlling efficacy of RNA silencing
EP2657248B1 (en) 2003-06-19 2017-03-22 Genzyme Corporation AAV virions with decreased immunoreactivity and uses therefor
US9441244B2 (en) 2003-06-30 2016-09-13 The Regents Of The University Of California Mutant adeno-associated virus virions and methods of use thereof
US7473531B1 (en) 2003-08-08 2009-01-06 Colora Corporation Pancreatic cancer targets and uses thereof
US20060107345A1 (en) 2003-09-30 2006-05-18 Nickolai Alexandrov Sequence-determined DNA fragments and corresponding polypeptides encoded thereby
EP3211085B1 (en) 2003-09-30 2021-03-31 The Trustees of The University of Pennsylvania Adeno-associated virus (aav) clades, sequences, vectors containing same, and uses therefor
CA2562024C (en) 2004-04-07 2014-05-27 Rinat Neuroscience Corp. Methods for treating bone cancer pain by administering a nerve growth factor antagonist
US20060171926A1 (en) 2004-04-30 2006-08-03 Passini Marco A Gene therapy for neurometabolic disorders
FR2874384B1 (en) 2004-08-17 2010-07-30 Genethon ADENO-ASSOCIATED VIRAL VECTOR FOR PRODUCING EXON JUMP IN A GENE ENCODING A PROTEIN WITH DISPENSABLE DOMAINS
JP4346526B2 (en) 2004-08-31 2009-10-21 株式会社東芝 Semiconductor integrated circuit device
AU2005282352B2 (en) 2004-09-09 2011-12-01 The General Hospital Corporation Modulating phosphatase activity in cardiac cells
EP1828390B1 (en) 2004-12-15 2012-06-13 The University Of North Carolina At Chapel Hill Chimeric vectors
JP4798663B2 (en) 2005-01-05 2011-10-19 塩野義製薬株式会社 Novel angiogenesis inhibitor
US20140199313A1 (en) 2005-03-02 2014-07-17 Metanomics Gmbh Process for the Production of Fine Chemicals
EP2194140A2 (en) 2005-03-02 2010-06-09 Metanomics GmbH Process for the production of fine chemicals
US20100196335A1 (en) 2005-04-29 2010-08-05 Samulski Richard J Methods and Compositions for Regulated Expressions of Nucleic Acid at Post-Transcriptional Level
US8283151B2 (en) 2005-04-29 2012-10-09 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Isolation, cloning and characterization of new adeno-associated virus (AAV) serotypes
WO2006138005A2 (en) 2005-05-10 2006-12-28 Monsanto Technology, Llc Genes and uses for plant improvement
US20100095387A1 (en) 2005-07-15 2010-04-15 Martin Smith Methods and reagents for screening new drugs and for treating ion pump associated disorders and diseases
EP1981548A4 (en) 2006-01-20 2010-03-24 Univ North Carolina Enhanced production of infectious parvovirus vectors in insect cells
WO2007089632A2 (en) 2006-01-27 2007-08-09 The University Of North Carolina At Chapel Hill Heparin and heparan sulfate binding chimeric vectors
HUE041928T2 (en) 2006-02-08 2019-06-28 Genzyme Corp Gene therapy for niemann-pick disease type a
EP1994043A4 (en) 2006-02-10 2009-05-20 Univ Cincinnati Phosphatase inhibitor protein-1 as a regulator of cardiac function
EP2007795B1 (en) 2006-03-30 2016-11-16 The Board Of Trustees Of The Leland Stanford Junior University Aav capsid proteins
ES2400235T3 (en) 2006-04-28 2013-04-08 The Trustees Of The University Of Pennsylvania AAV scalable production method
JP2009535339A (en) 2006-04-28 2009-10-01 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア Modified AAV vectors with reduced capsid immunogenicity and uses thereof
KR101589259B1 (en) 2006-06-21 2016-02-01 유니큐어 아이피 비.브이. -78 vectors with modified initiation codon for the translation of aav-rep78 useful for production of aav in insect cells
EP2125006B1 (en) 2007-01-18 2013-10-16 University of Missouri-Columbia Synthetic mini/micro-dystrophin genes to restore nnos to the sarcolemma
US20120322861A1 (en) 2007-02-23 2012-12-20 Barry John Byrne Compositions and Methods for Treating Diseases
US9725485B2 (en) 2012-05-15 2017-08-08 University Of Florida Research Foundation, Inc. AAV vectors with high transduction efficiency and uses thereof for gene therapy
US9611302B2 (en) 2007-04-09 2017-04-04 University Of Florida Research Foundation, Inc. High-transduction-efficiency RAAV vectors, compositions, and methods of use
EP2623605B1 (en) 2007-04-09 2018-11-28 University of Florida Research Foundation, Inc. RAAV vector compositions having tyrosine-modified capsid proteins and methods for use
KR100911624B1 (en) 2007-05-14 2009-08-12 연세대학교 산학협력단 Methods for Effectively Coexpressing ????? and ?????
US8664475B2 (en) 2007-09-18 2014-03-04 Basf Plant Science Gmbh Plants with increased yield
EP2058401A1 (en) 2007-10-05 2009-05-13 Genethon Widespread gene delivery to motor neurons using peripheral injection of AAV vectors
US10526627B2 (en) 2007-11-30 2020-01-07 Corn Products Development, Inc Method for producing high molecular weight reduced viscosity starch pastes
US20090221620A1 (en) 2008-02-20 2009-09-03 Celera Corporation Gentic polymorphisms associated with stroke, methods of detection and uses thereof
CN102007214A (en) 2008-02-20 2011-04-06 塞瑞斯公司 Nucleotide sequences and corresponding polypeptides conferring improved nitrogen use efficiency characteristics in plants
WO2009108274A2 (en) 2008-02-26 2009-09-03 The University Of North Carolina At Chapel Hill Methods and compositions for adeno-associated virus (aav) with hi loop mutations
CA2762118A1 (en) 2008-05-20 2010-01-28 Eos Neuroscience, Inc. Vectors for delivery of light-sensitive proteins and methods of use
JP2010002479A (en) 2008-06-18 2010-01-07 Crossfor:Kk Jewelry article for lens of eyeglasses and jewelry-installation tool for eyeglasses
US9415121B2 (en) 2008-12-19 2016-08-16 Nationwide Children's Hospital Delivery of MECP2 polynucleotide using recombinant AAV9
US8889641B2 (en) * 2009-02-11 2014-11-18 The University Of North Carolina At Chapel Hill Modified virus vectors and methods of making and using the same
US8679837B2 (en) 2009-04-02 2014-03-25 University Of Florida Research Foundation, Inc. Inducible system for highly efficient production of recombinant Adeno-associated virus (rAAV) vectors
HUE039345T2 (en) 2009-05-02 2018-12-28 Genzyme Corp Gene therapy for neurodegenerative disorders
WO2010138263A2 (en) 2009-05-28 2010-12-02 University Of Massachusetts Novel aav 's and uses thereof
US20120252877A1 (en) 2009-08-14 2012-10-04 Theramind Research, Llc Methods and compositions for treatment of tuberous sclerosis complex
EP2292781A1 (en) 2009-08-17 2011-03-09 Genethon Baculovirus-based production of biopharmaceuticals free of contaminating baculoviral virions
WO2011122950A1 (en) 2010-04-01 2011-10-06 Amsterdam Molecular Therapeutics (Amt) Ip B.V. Monomeric duplex aav vectors
EP2826860B1 (en) 2010-04-23 2018-08-22 University of Massachusetts CNS targeting AAV vectors and methods of use thereof
US8628966B2 (en) 2010-04-30 2014-01-14 City Of Hope CD34-derived recombinant adeno-associated vectors for stem cell transduction and systemic therapeutic gene transfer
US8927514B2 (en) 2010-04-30 2015-01-06 City Of Hope Recombinant adeno-associated vectors for targeted treatment
US9839696B2 (en) 2010-04-30 2017-12-12 City Of Hope Recombinant adeno-associated vectors for targeted treatment
JP5704361B2 (en) 2010-10-27 2015-04-22 学校法人自治医科大学 Adeno-associated virus virion for gene transfer into nervous system cells
ES2684307T3 (en) 2010-11-05 2018-10-02 The Board Of Trustees Of The Leland Stanford Junior University Stabilized opsin-type proteins of staggered function and methods of use thereof
WO2012064960A2 (en) 2010-11-10 2012-05-18 Fred Hutchinson Cancer Research Center Compositions and methods for generating adeno-associated viral vectors with undetectable capsid gene contamination
WO2012109570A1 (en) 2011-02-10 2012-08-16 The University Of North Carolina At Chapel Hill Viral vectors with modified transduction profiles and methods of making and using the same
US10392632B2 (en) 2011-02-14 2019-08-27 The Children's Hospital Of Philadelphia AAV8 vector with enhanced functional activity and methods of use thereof
WO2012159006A2 (en) 2011-05-18 2012-11-22 University Of Florida Research Foundation, Inc. Polypeptides and vectors for targeting her2/neu expressing cells and uses thereof
WO2013016315A1 (en) 2011-07-28 2013-01-31 Trustees Of Dartmouth College Methods for treating niemann-pick type c disease
CA3177742A1 (en) 2011-08-23 2013-02-28 Evogene Ltd. Isolated polynucleotides and polypeptides, and methods of using same for increasing plant yield and/or agricultural characteristics
WO2013078400A1 (en) 2011-11-22 2013-05-30 The Children's Hospital Of Philadelphia Virus vectors for highly efficient transgene delivery
FI124176B (en) 2011-12-01 2014-04-15 Helsingin Yliopisto Polypeptide, polynucleotide, expression cassette, vector, host cell, plant and uses
US20140350087A9 (en) 2012-03-22 2014-11-27 Halozyme, Inc. Oncovector Nucleic Acid Molecules and Methods of Use
CN104321432B (en) 2012-03-27 2018-08-10 库瑞瓦格股份公司 Include the artificial nucleic acid molecule of 5 ' TOP UTR
BR122021020419B1 (en) 2012-04-18 2023-01-17 The Children's Hospital Of Philadelphia ADENO-ASSOCIATED VIRUS (AAV) VECTOR COMPRISING A VP1 CAPSID PROTEIN, PHARMACEUTICAL COMPOSITIONS COMPRISING THE SAME, AND USES THEREOF
US20140162319A2 (en) 2012-05-02 2014-06-12 Sangeetha Hareendran Nucleotide sequences, methods, kit and a recombinant cell thereof
EP3470523A1 (en) 2012-05-09 2019-04-17 Oregon Health & Science University Adeno associated virus plasmids and vectors
US10294281B2 (en) 2012-05-15 2019-05-21 University Of Florida Research Foundation, Incorporated High-transduction-efficiency rAAV vectors, compositions, and methods of use
EP2492347A1 (en) 2012-05-22 2012-08-29 Laboratorios Del. Dr. Esteve, S.A. Methods for the production of vectors
CA2877428A1 (en) 2012-06-21 2013-12-27 Association Institut De Myologie Widespread gene delivery of gene therapy vectors
CN104619832B (en) 2012-07-06 2021-03-16 衣阿华大学研究基金会 Modified adeno-associated virus vector compositions
DK3444342T3 (en) 2012-07-11 2020-08-24 Sangamo Therapeutics Inc METHODS AND COMPOSITIONS FOR THE TREATMENT OF LYSOSOMAL DEPOSIT DISEASES
US9683268B2 (en) 2012-09-19 2017-06-20 Beth Israel Deaconess Viruses associated with immunodeficiency and enteropathy and methods using same
WO2014045674A1 (en) 2012-09-19 2014-03-27 国立大学法人東京大学 Dna exhibiting nerve activity-dependent promoter activity, and vector containing said dna
CN111621507A (en) 2012-09-28 2020-09-04 北卡罗来纳-查佩尔山大学 AAV vectors targeting oligodendrocytes
US9066966B2 (en) 2013-02-01 2015-06-30 Institut National De La Sante Et De La Recherche Medicale (Inserm) Methods and pharmaceutical compositions for the treatment of cardiomyopathy due to friedreich ataxia
DK2954051T3 (en) 2013-02-08 2019-07-08 Univ Pennsylvania MODIFIED KAPSID FOR TRANSFER FOR TREATMENT OF THE NETWORK
US10266845B2 (en) 2013-02-08 2019-04-23 The Trustees Of The University Of Pennsylvania Enhanced AAV-mediated gene transfer for retinal therapies
WO2014153083A1 (en) 2013-03-14 2014-09-25 The United States Of America, As Represented By The Secretary, Department Of Health & Human Services Compositions and methods for predicting age of onset of a lysosomal storage disease or a disease associated with a lysosomal defect
DK2968605T3 (en) 2013-03-15 2022-09-26 Univ North Carolina Chapel Hill METHODS AND COMPOSITIONS FOR DUAL GLYCAN BINDING AAV VECTORS
SG10201709552XA (en) 2013-05-21 2018-01-30 Univ Florida Capsid-modified, raav3 vector compositions and uses in gene therapy of human liver cancer
JP6600624B2 (en) 2013-05-31 2019-10-30 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア Adeno-associated virus mutant and method of use thereof
US9840719B2 (en) 2013-07-22 2017-12-12 The Children's Hospital Of Philadelphia Variant AAV and compositions, methods and uses for gene transfer to cells, organs and tissues
DE102013215817A1 (en) 2013-08-09 2015-02-12 Universitätsklinikum Hamburg-Eppendorf NEW PEPTIDES WITH SPECIFICITY FOR THE LUNG
EP3561062A1 (en) * 2013-09-13 2019-10-30 California Institute of Technology Selective recovery
US9710484B2 (en) * 2013-09-17 2017-07-18 Here Global B.V. Method and apparatus for associating physical locations to online entities
WO2015048534A1 (en) 2013-09-26 2015-04-02 University Of Florida Research Foundation, Inc. Synthetic combinatorial aav capsid library for targeted gene therapy
US20160256571A1 (en) 2013-09-30 2016-09-08 Sanofi Invention
EP3744730A1 (en) 2013-10-11 2020-12-02 Massachusetts Eye & Ear Infirmary Methods of predicting ancestral virus sequences and uses thereof
CN104592364B (en) * 2013-10-30 2018-05-01 北京大学 The adeno-associated virus of rite-directed mutagenesis and pointed decoration, its preparation method and application
GB201403684D0 (en) 2014-03-03 2014-04-16 King S College London Vector
WO2015127128A2 (en) 2014-02-19 2015-08-27 University Of Massachusetts Recombinant aavs having useful transcytosis properties
WO2015126972A1 (en) 2014-02-21 2015-08-27 University Of Florida Research Foundation, Inc. Methods and compositions for gene delivery to on bipolar cells
WO2015134643A1 (en) 2014-03-04 2015-09-11 University Of Florida Research Foundation, Inc. Improved raav vectors and methods for transduction of photoreceptors and rpe cells
WO2015164757A1 (en) 2014-04-25 2015-10-29 Oregon Health & Science University Methods of viral neutralizing antibody epitope mapping
SI3137497T1 (en) 2014-05-02 2021-08-31 Genzyme Corporation Aav vectors for retinal and cns gene therapy
AU2015265412B2 (en) 2014-05-28 2021-03-25 Evogene Ltd. Isolated polynucleotides, polypeptides and methods of using same for increasing abiotic stress tolerance, biomass and yield of plants
EP3151866B1 (en) 2014-06-09 2023-03-08 Voyager Therapeutics, Inc. Chimeric capsids
CA2953341C (en) 2014-06-25 2023-01-24 Acuitas Therapeutics Inc. Lipids and lipid nanoparticle formulations for delivery of nucleic acids
US9818383B2 (en) 2014-07-28 2017-11-14 University Of Connecticut Methods and systems for non-destructive analysis of objects and production of replica objects
CN113667696A (en) 2014-09-24 2021-11-19 希望之城 Adeno-associated virus vector variants for efficient genome editing and methods thereof
JP6842410B2 (en) 2014-10-03 2021-03-17 ユニバーシティ オブ マサチューセッツ AAV vector identified by a novel high efficiency library
CN107073051B (en) 2014-10-21 2021-08-24 马萨诸塞大学 Recombinant AAV variants and uses thereof
AU2016206624B2 (en) 2015-01-14 2020-08-06 The University Of North Carolina At Chapel Hill Methods and compositions for targeted gene transfer
EP3245221A4 (en) 2015-01-16 2018-06-13 Voyager Therapeutics, Inc. Central nervous system targeting polynucleotides
WO2016126857A1 (en) 2015-02-03 2016-08-11 University Of Florida Research Foundation, Inc. Recombinant aav1, aav5, and aav6 capsid mutants and uses thereof
GB201502306D0 (en) 2015-02-12 2015-04-01 Hansa Medical Ab Protein
GB201502305D0 (en) 2015-02-12 2015-04-01 Hansa Medical Ab Protein
WO2016134338A1 (en) 2015-02-19 2016-08-25 University Of Florida Research Foundation, Inc. Recombinant aav vectors for gene therapy of human hematopoietic disorders
EP3265178A4 (en) 2015-03-02 2018-09-05 Adverum Biotechnologies, Inc. Compositions and methods for intravitreal delivery of polynucleotides to retinal cones
EP3273999A1 (en) 2015-03-23 2018-01-31 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods and pharmaceutical composition for the treatment and the prevention of neurological phenotype associated with friedreich ataxia
MX2017012097A (en) 2015-03-24 2018-06-15 Univ California Adeno-associated virus variants and methods of use thereof.
US10081659B2 (en) 2015-04-06 2018-09-25 The United States Of America, As Represented By The Secretary, Dept. Of Health And Human Services Adeno-associated vectors for enhanced transduction and reduced immunogenicity
ES2755725T3 (en) 2015-04-08 2020-04-23 Us Health Viral gene therapy as treatment for a cholesterol storage disease or disorder
US11020443B2 (en) 2015-04-23 2021-06-01 University Of Massachusetts Modulation of AAV vector transgene expression
US11046955B2 (en) 2015-04-24 2021-06-29 University Of Massachusetts Modified AAV constructs and uses thereof
US11104917B2 (en) 2015-05-08 2021-08-31 Children's Medical Research Institute Promoters for expression of heterologous genes
GB201508026D0 (en) * 2015-05-11 2015-06-24 Ucl Business Plc Capsid
GB201508025D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Fabry disease gene therapy
JP6805174B2 (en) 2015-05-12 2020-12-23 アメリカ合衆国 AAV isolates and fusion proteins containing nerve growth factor signal peptides and parathyroid hormone
WO2017015102A1 (en) 2015-07-17 2017-01-26 The Trustees Of The University Of Pennsylvania Compositions and methods for achieving high levels of transduction in human liver cells
WO2017013648A1 (en) 2015-07-19 2017-01-26 Yeda Research And Development Co. Ltd. SELECTIVE INHIBITORS OF Alpha2-CONTAINING ISOFORMS OF Na,K-ATPase AND USE THEREOF FOR REDUCTION OF INTRAOCULAR PRESSURE
WO2017019876A1 (en) 2015-07-28 2017-02-02 University Of Massachusetts Transgenic expression of dnase i in vivo delivered by an adeno-associated virus vector
LT3872085T (en) 2015-07-30 2023-05-10 Massachusetts Eye & Ear Infirmary Ancestral aav sequences and uses thereof
JP7064214B2 (en) 2015-09-28 2022-05-10 ザ・ユニヴァーシティ・オヴ・ノース・キャロライナ・アト・チャペル・ヒル Methods and Compositions for Antibodies Avoidant Viral Vectors
WO2017066764A2 (en) 2015-10-16 2017-04-20 William Marsh Rice University Modification of n-terminal region of capsid proteins for enhanced properties of adeno-associated viruses
CA3002980A1 (en) 2015-10-22 2017-04-27 University Of Massachusetts Prostate-targeting adeno-associated virus serotype vectors
WO2017077451A1 (en) 2015-11-05 2017-05-11 Bamboo Therapeutics, Inc. Modified friedreich ataxia genes and vectors for gene therapy
CA3006309A1 (en) 2015-12-01 2017-06-08 Spark Therapeutics, Inc. Scalable methods for producing recombinant adeno-associated viral (aav) vector in serum-free suspension cell culture system suitable for clinical use
US10406244B2 (en) 2015-12-02 2019-09-10 The Board Of Trustees Of The Leland Stanford Junior University AAV vectors with expanded packaging capacity
US10385320B2 (en) 2015-12-02 2019-08-20 The Board Of Trustees Of The Leland Stanford Junior University Recombinant adeno-associated virus capsids with enhanced human skeletal muscle tropism
SG11201804713VA (en) 2015-12-11 2018-07-30 California Inst Of Techn TARGETING PEPTIDES FOR DIRECTING ADENO-ASSOCIATED VIRUSES (AAVs)
MX2018007237A (en) 2015-12-14 2018-12-11 Univ North Carolina Chapel Hill Modified capsid proteins for enhanced delivery of parvovirus vectors.
EP4094780A3 (en) 2016-02-12 2023-02-08 University of Massachusetts Anti-angiogenic mirna therapeutics for inhibiting corneal neovascularization
CN109415704B (en) 2016-02-16 2022-02-25 利兰斯坦福初级大学董事会 Novel recombinant adeno-associated virus capsids resistant to pre-existing human neutralizing antibodies
AU2017223465A1 (en) 2016-02-22 2018-08-09 The University Of North Carolina At Chapel Hill AAV-IDUA vector for treatment of MPS I-associated blindness
JP7140683B2 (en) 2016-04-15 2022-09-21 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア Novel AAV8 Mutant Capsids and Compositions Containing Them
US11866462B2 (en) 2016-05-04 2024-01-09 Oregon Health & Science University Recombinant adeno-associated viral vectors
NZ748395A (en) 2016-05-13 2019-12-20 4D Molecular Therapeutics Inc Adeno-associated virus variant capsids and methods of use thereof
KR20240056729A (en) 2016-05-18 2024-04-30 보이저 테라퓨틱스, 인크. Modulatory polynucleotides
CN109790549A (en) 2016-07-21 2019-05-21 星火治疗有限公司 Produce recombinant adeno-associated virus (rAAV) carrier of the expansible high absorption method of high yield recombinant adeno-associated virus (rAAV) carrier and its generation
JP6994018B2 (en) 2016-07-26 2022-01-14 バイオマリン ファーマシューティカル インコーポレイテッド New adeno-associated virus capsid protein
CA3033125A1 (en) 2016-08-16 2018-02-22 The University Of North Carolina At Chapel Hill Methods and compositions for targeted gene transfer
IL265045B2 (en) 2016-09-08 2023-09-01 Bluebird Bio Inc Pd-1 homing endonuclease variants, compositions, and methods of use
US11078238B2 (en) 2016-09-29 2021-08-03 University Of Florida Research Foundation, Incorporated AAVrh.10 variants with host antibody escape capabilities and altered tissue targeting properties
EP3528785A4 (en) 2016-10-19 2020-12-02 Adverum Biotechnologies, Inc. Modified aav capsids and uses thereof
US20230048025A1 (en) 2016-12-22 2023-02-16 Oregon Health & Science University Adeno associated viral vectors
AU2018221738A1 (en) 2017-02-15 2019-08-22 The University Of North Carolina At Chapel Hill Methods and compositions for gene transfer across the vasculature
JP7455579B2 (en) 2017-02-28 2024-03-26 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア Novel adeno-associated virus (AAV) clade F vectors and their uses
IL268891B1 (en) 2017-03-15 2024-05-01 Univ North Carolina Chapel Hill Polyploid adeno-associated virus vectors and methods of making and using the same
US10550405B2 (en) 2017-03-15 2020-02-04 The University Of North Carolina At Chapel Hill Rational polyploid adeno-associated virus vectors and methods of making and using the same
WO2018204764A1 (en) 2017-05-05 2018-11-08 Camp4 Therapeutics Corporation Identification and targeted modulation of gene signaling networks
JP7330899B2 (en) 2017-05-10 2023-08-22 マサチューセッツ アイ アンド イヤー インファーマリー Methods and compositions for altering viral assembly-activating protein (AAP) dependence
EA201992882A1 (en) 2017-06-06 2020-05-25 Юниверсити Оф Массачусеттс AAV SELF-ADJUSTABLE VECTORS BASED ON SAFE MeCP2 EXPRESSION AT PETTA SYNDROME
WO2018237066A1 (en) 2017-06-20 2018-12-27 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Codon-optimized human npc1 genes for the treatment of niemann-pick type c1 deficiency and related conditions
WO2019006418A2 (en) 2017-06-30 2019-01-03 Intima Bioscience, Inc. Adeno-associated viral vectors for gene therapy
US20210228738A1 (en) 2017-07-17 2021-07-29 INSERM (Institut National de la Santé et de la Recherche Médicale) Compositions and methods for increasing or enhancing transduction of gene therapy vectors and for removing or reducing immunoglobulins
WO2019025984A1 (en) 2017-07-31 2019-02-07 Reflection Biotechnologies Limited Cellular models of and therapies for ocular diseases
MX2020006764A (en) 2018-01-17 2021-01-08 Meiragtx Uk Ii Ltd A modified raav capsid protein for gene therapy.
MX2020008932A (en) 2018-02-27 2020-10-01 Univ Pennsylvania Novel adeno-associated virus (aav) vectors, aav vectors having reduced capsid deamidation and uses therefor.
CA3091806A1 (en) 2018-02-27 2019-09-06 The Trustees Of The University Of Pennsylvania Novel adeno-associated virus (aav) vectors, aav vectors having reduced capsid deamidation and uses therefor
EP3759218A4 (en) 2018-02-28 2021-12-08 The University of North Carolina at Chapel Hill Methods and compositions for antibody-evading virus vectors
US20210010028A1 (en) 2018-03-06 2021-01-14 Voyager Therapeutics, Inc. Insect cell manufactured partial self-complementary aav genomes
CA3092871A1 (en) 2018-03-06 2019-09-12 University Of Florida Research Foundation, Incorporated Aav chimeras
WO2019178412A1 (en) 2018-03-16 2019-09-19 Research Institute At Nationwide Children's Hospital Increasing tissue specific gene delivery by capsid modification
EP3774854A1 (en) 2018-04-03 2021-02-17 Stridebio, Inc. Antibody-evading virus vectors
CN112533644A (en) 2018-04-03 2021-03-19 斯特里迪比奥公司 Viral vectors targeting ocular tissues
CN112543766A (en) * 2018-04-03 2021-03-23 斯特里迪比奥公司 Antibody-evasive viral vectors
CN113302201A (en) 2018-11-21 2021-08-24 斯特里迪比奥公司 Recombinant viral vectors and nucleic acids for producing the same
EP4269579A3 (en) 2019-01-04 2024-01-24 Ultragenyx Pharmaceutical Inc. Gene therapy constructs for treating wilson disease
EA202192501A1 (en) 2019-03-21 2021-12-16 Страйдбайо, Инк. RECOMBINANT ADENO-ASSOCIATED VIRAL VECTORS
JP2022531809A (en) 2019-05-14 2022-07-11 デューク ユニバーシティ Compositions and Methods for the Treatment of ATPase-mediated Diseases
US20240131185A1 (en) 2019-10-17 2024-04-25 Stridebio, Inc. Aav transfer cassette
WO2021076925A1 (en) 2019-10-17 2021-04-22 Stridebio, Inc. Adeno-associated viral vectors for treatment of niemann-pick disease type c
IL300717A (en) 2020-08-19 2023-04-01 Sarepta Therapeutics Inc Adeno-associated virus vectors for treatment of rett syndrome

Also Published As

Publication number Publication date
EP3941929A1 (en) 2022-01-26
JP2022525955A (en) 2022-05-20
US20220064675A1 (en) 2022-03-03
US11981914B2 (en) 2024-05-14
MX2021011468A (en) 2021-12-15
WO2020191300A1 (en) 2020-09-24
SG11202110146VA (en) 2021-10-28
AU2020241888A1 (en) 2021-09-30
CA3133453A1 (en) 2020-09-24
TW202102525A (en) 2021-01-16
AR118465A1 (en) 2021-10-06
CN113727992A (en) 2021-11-30
KR20220011616A (en) 2022-01-28
EA202192501A1 (en) 2021-12-16
US20240067986A1 (en) 2024-02-29
IL286464A (en) 2021-12-01
CO2021013878A2 (en) 2022-01-17

Similar Documents

Publication Publication Date Title
BR112021018776A2 (en) Recombinant adeno-associated virus vectors
MX2019009720A (en) Methods and compositions for gene transfer across the vasculature.
MX2020010466A (en) Antibody-evading virus vectors.
WO2017058892A3 (en) Methods and compositions for antibody-evading virus vectors
PH12020550094A1 (en) Adeno-associated virus variant capsids and methods of use thereof
MX2020010464A (en) Antibody-evading virus vectors.
MX2020008933A (en) Novel adeno-associated virus (aav) vectors, aav vectors having reduced capsid deamidation and uses therefor.
MX2020010465A (en) Virus vectors for targeting ophthalmic tissues.
PH12018502387A1 (en) Adeno-associated virus variant capsids and methods of use thereof
MX2020008932A (en) Novel adeno-associated virus (aav) vectors, aav vectors having reduced capsid deamidation and uses therefor.
PH12020550706A1 (en) Adeno-associated virus variant capsids and use for inhibiting angiogenesis
BR112019002904A2 (en) methods and compositions for directed gene transfer
AR080229A1 (en) FIBRONECTINE ARMAZON DOMAIN PROTEINS THAT JOIN IL -23 (INTERLEUCINE 23)
EA201200515A1 (en) POLYPEPTIDES AND THEIR APPLICATION
EA202091352A1 (en) GENOTHERAPY OF MUCOPOLYSACCHARIDOSIS IIIB
BR0012919A (en) Vaccine for use in the prophylaxis and / or treatment of white spot syndrome in crustaceans, structural protein derived from white point syndrome virus, nucleic acid sequence, use of a structural protein, pharmaceutical composition, antibodies, vaccine or pharmaceutical formulation, and, diagnostic kit for wssv detection
BR112021021792A2 (en) Useful compositions for treatment of pomp disease
CL2023001876A1 (en) Viral capsid proteins with specificity for cardiac tissue cells
EA202092065A1 (en) EXPRESSION OF PNEUMOCOCCAL SURFACE PROTEIN A (PspA)
EA202092046A1 (en) NEW VECTORS BASED ON THE ADENO-ASSOCIATED VIRUS (AAV), AAV VECTORS WITH REDUCED CAPSID DESAMIDATION, AS WELL AS THEIR APPLICATION
EA202092363A1 (en) VIRAL VECTORS ESCAPING ANTIBODIES
EA202092353A1 (en) VIRAL VECTORS ESCAPING ANTIBODIES
EA202092015A1 (en) NEW VECTORS BASED ON THE ADENO-ASSOCIATED VIRUS (AAV), AAV VECTORS WITH REDUCED CAPSID DESAMIDATION, AND THEIR APPLICATION
EA202192973A1 (en) COMPOSITIONS USEFUL IN THE TREATMENT OF POMPE DISEASE
MY186651A (en) Adeno-associated virus variant capsids and methods of use thereof

Legal Events

Date Code Title Description
B25A Requested transfer of rights approved

Owner name: GINKGO BIOWORKS, INC. (US)